Trial Outcomes & Findings for Burden of Disease Study In Patients With Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP) orTransthyretin Cardiomyopathy (TTR-CM) And Caregivers (NCT NCT01604122)
NCT ID: NCT01604122
Last Updated: 2023-04-06
Results Overview
Main characteristics included were education level and employment status which were asked from all participants and caregivers. Type of job (full-time, part-time) was asked only from those participants and caregivers who provided their employment status as employed. Those who were unemployed reported their cause of unemployment, whether it was due to ATTR or not.
COMPLETED
1 participants
Baseline (Day 1)
2023-04-06
Participant Flow
Participant milestones
| Measure |
Participants Diagnosed With ATTR
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
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|---|---|---|
|
Overall Study
STARTED
|
60
|
32
|
|
Overall Study
COMPLETED
|
60
|
32
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Burden of Disease Study In Patients With Transthyretin Familial Amyloidosis Polyneuropathy (TTR-FAP) orTransthyretin Cardiomyopathy (TTR-CM) And Caregivers
Baseline characteristics by cohort
| Measure |
Participants Diagnosed With ATTR
n=60 Participants
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
n=32 Participants
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
|
Total
n=92 Participants
Total of all reporting groups
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|---|---|---|---|
|
Age, Continuous
|
58.4 years
STANDARD_DEVIATION 12.0 • n=5 Participants
|
55.9 years
STANDARD_DEVIATION 12.8 • n=7 Participants
|
57.5 years
STANDARD_DEVIATION 12.2 • n=5 Participants
|
|
Sex: Female, Male
Female
|
13 Participants
n=5 Participants
|
22 Participants
n=7 Participants
|
35 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
47 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
57 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline (Day 1)Population: All participants who were enrolled in the study and completed the survey. Here, 'n' signifies those participants who were evaluable for specific category for each arm respectively.
Main characteristics included were education level and employment status which were asked from all participants and caregivers. Type of job (full-time, part-time) was asked only from those participants and caregivers who provided their employment status as employed. Those who were unemployed reported their cause of unemployment, whether it was due to ATTR or not.
Outcome measures
| Measure |
Participants Diagnosed With ATTR
n=60 Participants
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
n=32 Participants
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
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|---|---|---|
|
Demographical Characteristics of Participants
Employment Status: Currently Employed (n= 60, 32)
|
20 participants
|
11 participants
|
|
Demographical Characteristics of Participants
Employment Status:Not Currently Employed (n=60,32)
|
39 participants
|
19 participants
|
|
Demographical Characteristics of Participants
Employment Status: Skipped (n= 60, 32)
|
1 participants
|
1 participants
|
|
Demographical Characteristics of Participants
Employment Status: Not Asked (n= 60, 32)
|
0 participants
|
1 participants
|
|
Demographical Characteristics of Participants
Employment Type: Full-Time (n= 20, 11)
|
11 participants
|
9 participants
|
|
Demographical Characteristics of Participants
Employment Type: Part-Time (n= 20, 11)
|
9 participants
|
2 participants
|
|
Demographical Characteristics of Participants
Unemployed: Due to ATTR (n= 39, 19)
|
25 participants
|
0 participants
|
|
Demographical Characteristics of Participants
Unemployed: Not due to ATTR (n= 39, 19)
|
13 participants
|
0 participants
|
|
Demographical Characteristics of Participants
Unemployed: Skipped (n= 39, 19)
|
1 participants
|
0 participants
|
|
Demographical Characteristics of Participants
Unemployed: Not Asked (n= 39, 19)
|
0 participants
|
19 participants
|
|
Demographical Characteristics of Participants
Education Level: Lower Than High School (n=60,32)
|
6 participants
|
3 participants
|
|
Demographical Characteristics of Participants
Education Level: High School/Equivalent(n=60,32)
|
8 participants
|
6 participants
|
|
Demographical Characteristics of Participants
Education Level: Associate Degree(n=60,32)
|
12 participants
|
3 participants
|
|
Demographical Characteristics of Participants
Education Level: College Degree (n= 60, 32)
|
14 participants
|
10 participants
|
|
Demographical Characteristics of Participants
Education Level: Professional or Graduate(n=60,32)
|
20 participants
|
7 participants
|
|
Demographical Characteristics of Participants
Education Level: Skipped (n= 60, 32)
|
0 participants
|
2 participants
|
|
Demographical Characteristics of Participants
Education Level: Not Asked (n= 60, 32)
|
0 participants
|
1 participants
|
PRIMARY outcome
Timeframe: Baseline (Day 1)Population: All participants who were enrolled in the study and completed the survey.
Duration of disease was defined as the time from diagnosis of disease until baseline visit. This outcome measure was planned to be assessed for reporting arm of participants diagnosed with ATTR.
Outcome measures
| Measure |
Participants Diagnosed With ATTR
n=60 Participants
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
|
|---|---|---|
|
Disease Characteristics of Participants: Disease Duration
|
5.6 years
Full Range 4.0 • Interval 0.0 to 16.1
|
—
|
PRIMARY outcome
Timeframe: Baseline (Day 1)Population: All participants who were enrolled in the study and completed the survey.
Genetic mutation leads to misfolding of protein transthyretin (TTR) which results in ATTR. In this outcome, number of participants with each type of resulted mutation type (Val30Met, wild type TTR, Phe64Leu, Ser77Tyr, Thr60Ala or other than these) were reported. This outcome was planned to be assessed for reporting arm of participants diagnosed with ATTR.
Outcome measures
| Measure |
Participants Diagnosed With ATTR
n=60 Participants
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
|
|---|---|---|
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Disease Characteristics of Participants: Mutation Type
Val30Met
|
18 participants
|
—
|
|
Disease Characteristics of Participants: Mutation Type
Wild Type TTR
|
2 participants
|
—
|
|
Disease Characteristics of Participants: Mutation Type
Phe64Leu
|
4 participants
|
—
|
|
Disease Characteristics of Participants: Mutation Type
Ser77Tyr
|
3 participants
|
—
|
|
Disease Characteristics of Participants: Mutation Type
Thr60Ala
|
11 participants
|
—
|
|
Disease Characteristics of Participants: Mutation Type
Other
|
5 participants
|
—
|
|
Disease Characteristics of Participants: Mutation Type
Unknown
|
17 participants
|
—
|
PRIMARY outcome
Timeframe: Baseline (Day 1)Population: All participants who were enrolled in the study and completed the survey.
TTR protein is primarily synthesized in the liver. Liver transplantation was considered as one of the measure to eliminate the main source of variant TTR. In the study, participants who were diagnosed with ATTR were asked for their liver transplantation status (whether they had transplantation or not). In this outcome measure, number of participants with liver transplant status were reported. This outcome was planned to be assessed for reporting arm of participants diagnosed with ATTR.
Outcome measures
| Measure |
Participants Diagnosed With ATTR
n=60 Participants
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
|
|---|---|---|
|
Disease Characteristics of Participants: Liver Transplantation Status
With Liver Transplantation
|
31 participants
|
—
|
|
Disease Characteristics of Participants: Liver Transplantation Status
No Liver Transplantation
|
29 participants
|
—
|
PRIMARY outcome
Timeframe: Baseline (Day 1)Population: All participants who were enrolled in the study and completed the survey.
Family history of participants diagnosed with ATTR was assessed to determine whether family history of ATTR was a significant risk factor for ATTR or not. This outcome was planned to be assessed for reporting arm of participants diagnosed with ATTR.
Outcome measures
| Measure |
Participants Diagnosed With ATTR
n=60 Participants
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
|
|---|---|---|
|
Disease Characteristics of Participants: Number of Participants With Family History of ATTR
|
51 participants
|
—
|
PRIMARY outcome
Timeframe: Baseline (Day 1)Population: All participants who were included in the study and completed the survey.
Mobility, i.e., ability to walk was assessed as a part of loss of functioning in the participants diagnosed with ATTR. In this outcome, number of participants with their different mobility status along with the use of mobility aids (able to walk normally, some problems with feet but able to walk without difficulty, some difficulty walking but can walk without help, confined to bed all the time, need 1 cane or crutch to walk, need 2 canes/crutches or a walker to walk) were reported.
Outcome measures
| Measure |
Participants Diagnosed With ATTR
n=60 Participants
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
|
|---|---|---|
|
Disease Characteristics of Participants: Mobility Status
Able to Walk Normally
|
15 participants
|
—
|
|
Disease Characteristics of Participants: Mobility Status
Some Problems: Able to Walk Without Difficulty
|
16 participants
|
—
|
|
Disease Characteristics of Participants: Mobility Status
Some Difficulty: Can Walk Without Help
|
14 participants
|
—
|
|
Disease Characteristics of Participants: Mobility Status
Need 1 Cane or Crutch to Walk
|
6 participants
|
—
|
|
Disease Characteristics of Participants: Mobility Status
Need 2 Canes/Crutches or a Walker to Walk
|
9 participants
|
—
|
|
Disease Characteristics of Participants: Mobility Status
Confined to bed all the Time
|
0 participants
|
—
|
PRIMARY outcome
Timeframe: Baseline (Day 1)Population: All participants who were enrolled in the study and completed the survey. Here, 'n' signifies those participants who were evaluable for specific component for each arm respectively.
SF-12 was a patient reported outcome survey that represented overall health status by measuring 8 health-related aspects of an individual: Body pain, general mental health, perception of general health, physical functioning, role limitations caused by mental condition, role limitations caused by a physical condition, social functioning, and vitality. The score range for each of the 8 health aspects was from 0 (poor health) to 100 (better health), higher scores indicating good health condition. Responses on the SF-12 were also used to calculate 2 summary scores: Physical component score (PCS) and mental component score (MCS). The score range for each of these 2 summary scores was from 0 (poor health) to 100 (better health), where 100 indicated good health condition.
Outcome measures
| Measure |
Participants Diagnosed With ATTR
n=60 Participants
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
n=32 Participants
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
|
|---|---|---|
|
12-Item Short-Form Health Survey (SF-12) Scores
Physical Health Summary (n= 56, 28)
|
33.6 units on a scale
Standard Deviation 11.5
|
42.2 units on a scale
Standard Deviation 13.7
|
|
12-Item Short-Form Health Survey (SF-12) Scores
Mental Health Summary (n= 56, 28)
|
47.1 units on a scale
Standard Deviation 10.8
|
39.3 units on a scale
Standard Deviation 11.1
|
|
12-Item Short-Form Health Survey (SF-12) Scores
Physical Functioning (n= 58, 29)
|
32.2 units on a scale
Standard Deviation 11.0
|
43.1 units on a scale
Standard Deviation 13.7
|
|
12-Item Short-Form Health Survey (SF-12) Scores
Role-Physical (n= 56, 28)
|
33.7 units on a scale
Standard Deviation 10.7
|
39.1 units on a scale
Standard Deviation 13.1
|
|
12-Item Short-Form Health Survey (SF-12) Scores
Bodily Pain (n= 57, 29)
|
43.0 units on a scale
Standard Deviation 13.3
|
39.5 units on a scale
Standard Deviation 15.3
|
|
12-Item Short-Form Health Survey (SF-12) Scores
General Health (n= 59, 29)
|
37.2 units on a scale
Standard Deviation 12.0
|
39.8 units on a scale
Standard Deviation 13.5
|
|
12-Item Short-Form Health Survey (SF-12) Scores
Vitality (n= 56, 29)
|
41.1 units on a scale
Standard Deviation 10.6
|
45.7 units on a scale
Standard Deviation 11.5
|
|
12-Item Short-Form Health Survey (SF-12) Scores
Social Functioning (n= 57, 29)
|
41.0 units on a scale
Standard Deviation 11.6
|
39.5 units on a scale
Standard Deviation 13.8
|
|
12-Item Short-Form Health Survey (SF-12) Scores
Role-Emotional (n= 57, 29)
|
39.5 units on a scale
Standard Deviation 13.9
|
34.3 units on a scale
Standard Deviation 13.6
|
|
12-Item Short-Form Health Survey (SF-12) Scores
Mental Health (n= 57, 29)
|
48.2 units on a scale
Standard Deviation 10.2
|
41.8 units on a scale
Standard Deviation 11.4
|
PRIMARY outcome
Timeframe: Baseline (Day 1)Population: All participants who were enrolled in the study and completed the survey. Here, 'N' (number of participants analyzed) signifies those participants who were evaluable for this measure.
HADS: participant rated 14-item questionnaire with 2 subscales; HADS-anxiety scale (HADS-A) and HADS-depression scale (HADS-D). HADS-A assesses state of generalized anxiety (anxious mood, restlessness, anxious thoughts, panic attacks); HADS-D assesses state of lost interest and diminished pleasure response (lowering of hedonic tone). Each subscale comprised of 7 items and the participant responds as to how each item applies to him/her over the past week prior to baseline visit, on 4-point response scale. Separate scores were calculated for anxiety and depression with score ranges from 0 (no presence of anxiety or depression) to 3 (severe feeling of anxiety or depression). Total score range was from 0 to 21 for each subscale; higher score indicating greater severity of anxiety and depression symptoms.
Outcome measures
| Measure |
Participants Diagnosed With ATTR
n=55 Participants
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
n=29 Participants
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
|
|---|---|---|
|
Hospital Anxiety and Depression Scale (HADS): Depression and Anxiety Subscale Scores
Depression Sub-Scale
|
5.9 units on a scale
Standard Deviation 3.4
|
8.1 units on a scale
Standard Deviation 5.3
|
|
Hospital Anxiety and Depression Scale (HADS): Depression and Anxiety Subscale Scores
Anxiety Sub-Scale
|
5.9 units on a scale
Standard Deviation 3.9
|
10.8 units on a scale
Standard Deviation 4.5
|
PRIMARY outcome
Timeframe: Baseline (Day 1)Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure.
EQ-5D-3L: participant rated questionnaire to assess generic health status in two parts: single utility score and visual analog scale. For utility score, participants rated their current health state on 5 dimensions: mobility, self-care, usual activities, pain and discomfort, and anxiety and depression with each dimension having three levels of function: 1 indicates no problem; 2 indicates some problem; 3 indicates extreme problem. Scoring formula developed by EuroQol Group assigns a utility value for each domain in the profile. Score was transformed and results in a total score range of 0.05 to 1.00; higher scores indicating a better health state.
Outcome measures
| Measure |
Participants Diagnosed With ATTR
n=57 Participants
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
n=28 Participants
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
|
|---|---|---|
|
Euro Quality of Life (EQ-5D-3L)- Health State Profile Utility Score
|
0.74 units on a scale
Standard Deviation 0.20
|
0.65 units on a scale
Standard Deviation 0.33
|
PRIMARY outcome
Timeframe: Baseline (Day 1)Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure.
EQ-5D: participant rated questionnaire to assess generic health status in two parts: single utility score and visual analog scale. The VAS component rated the current health state on a scale ranging from 0 (worst imaginable health state) to 100 (best imaginable health state); higher scores indicating a better health state.
Outcome measures
| Measure |
Participants Diagnosed With ATTR
n=55 Participants
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
n=28 Participants
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
|
|---|---|---|
|
Euro Quality of Life (EQ-5D-3L)- Visual Analog Scale (VAS) Score
|
58.7 units on a scale
Standard Deviation 19.8
|
64.2 units on a scale
Standard Deviation 28.1
|
PRIMARY outcome
Timeframe: Baseline (Day 1)Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure.
The WPAI assesses work productivity and impairment. It was a 6-item questionnaire used to assess the degree to which a specified health problem affected work productivity and regular activities over the past 7 days prior to baseline visit. The questionnaire asked about current employment status, hours worked, hours missed from work and degree to which a specified health problem (ATTR) or caregiving affected work productivity and regular activities. Percentage of work time missed of participants were recorded and reported.
Outcome measures
| Measure |
Participants Diagnosed With ATTR
n=18 Participants
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
n=9 Participants
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
|
|---|---|---|
|
Work Productivity and Activity Impairment- Specific Health Version (WPAI-SH): Percent of Work Time Missed
|
2 percentage of work time missed
Full Range 26.0 • Interval 0.0 to 79.0
|
0 percentage of work time missed
Full Range 21.8 • Interval 0.0 to 57.0
|
PRIMARY outcome
Timeframe: Baseline (Day 1)Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure.
The WPAI assesses work productivity and impairment. It was a 6-item questionnaire used to assess the degree to which a specified health problem affected work productivity and regular activities over the past 7 days prior to baseline visit. The questionnaire asks about current employment status, hours worked, hours missed from work and degree to which a specified health problem (ATTR) or caregiving affected work productivity and regular activities. Component scores included percent work time missed due to the health problem; percent impairment while working due to problem; percent overall work impairment due to problem; and percent activity impairment due to problem. The computed percentage range for each sub-scale was from 0-100, where higher numbers indicating greater impairment and less productivity.
Outcome measures
| Measure |
Participants Diagnosed With ATTR
n=18 Participants
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
n=8 Participants
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
|
|---|---|---|
|
Work Productivity and Activity Impairment- Specific Health Version: Percent Impairment While Working
|
32.8 units on a scale
Standard Deviation 32.7
|
36.3 units on a scale
Standard Deviation 31.6
|
PRIMARY outcome
Timeframe: Baseline (Day 1)Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure.
The WPAI assesses work productivity and impairment. It was a 6-item questionnaire used to assess the degree to which a specified health problem affected work productivity and regular activities over the past 7 days prior to baseline visit. The questionnaire asked about current employment status, hours worked, hours missed from work and degree to which a specified health problem (ATTR) or caregiving affected work productivity and regular activities. Component scores included percent work time missed due to the health problem; percent impairment while working due to problem; percent overall work impairment due to problem; and percent activity impairment due to problem. The computed percentage range for each sub-scale was from 0-100, where higher numbers indicating greater impairment and less productivity.
Outcome measures
| Measure |
Participants Diagnosed With ATTR
n=18 Participants
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
n=8 Participants
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
|
|---|---|---|
|
Work Productivity and Activity Impairment- Specific Health Version: Percent Overall Work Impairment
|
37.3 units on a scale
Standard Deviation 36.1
|
40.4 units on a scale
Standard Deviation 35.6
|
PRIMARY outcome
Timeframe: Baseline (Day 1)Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure.
The WPAI assesses work productivity and impairment. It was a 6-item questionnaire used to assess the degree to which a specified health problem affected work productivity and regular activities over the past 7 days prior to baseline visit. The questionnaire asks about current employment status, hours worked, hours missed from work and degree to which a specified health problem (ATTR) or caregiving affected work productivity and regular activities. Component scores included percent work time missed due to the health problem; percent impairment while working due to problem; percent overall work impairment due to problem; and percent activity impairment due to problem. The computed percentage range for each sub-scale was from 0-100, where higher numbers indicating greater impairment and less productivity.
Outcome measures
| Measure |
Participants Diagnosed With ATTR
n=55 Participants
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
n=17 Participants
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
|
|---|---|---|
|
Work Productivity and Activity Impairment- Specific Health Version: Percent Activity Impairment
|
49.8 units on a scale
Standard Deviation 30.5
|
41.2 units on a scale
Standard Deviation 30.4
|
PRIMARY outcome
Timeframe: Baseline (Day 1)Population: All participants who were enrolled in the study and completed the survey. Here, 'n' signifies those participants who were evaluable for specific category for each arm respectively.
Healthcare resources use survey of participants diagnosed with ATTR and caregivers was assessed by questions concerning a variety of different types of treatment and resources including outpatient visits to healthcare providers, hospitalizations, emergency/urgent care visits, symptomatic treatments, and out-of-pocket costs (for example, costs of travel to receive care).
Outcome measures
| Measure |
Participants Diagnosed With ATTR
n=60 Participants
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
n=32 Participants
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
|
|---|---|---|
|
Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers
Primary Care Provider/Family Doctor (n=27, 12)
|
2.0 outpatient visits
Standard Deviation 1.5
|
1.8 outpatient visits
Standard Deviation 1.5
|
|
Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers
Neurologist (n=12, 6)
|
1.4 outpatient visits
Standard Deviation 0.8
|
1.2 outpatient visits
Standard Deviation 0.4
|
|
Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers
Cardiologist (n=17, 9)
|
2.2 outpatient visits
Standard Deviation 2.7
|
1.1 outpatient visits
Standard Deviation 0.3
|
|
Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers
Gastroenterologist (n=9, 3)
|
1.3 outpatient visits
Standard Deviation 0.5
|
1.3 outpatient visits
Standard Deviation 0.6
|
|
Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers
Hematologist (n=5, 4)
|
2.8 outpatient visits
Standard Deviation 0.8
|
1.8 outpatient visits
Standard Deviation 1.5
|
|
Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers
Liver Transplant Surgeon (n=6, 5)
|
1.2 outpatient visits
Standard Deviation 0.4
|
1.0 outpatient visits
Standard Deviation 0.0
|
|
Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers
Nurse/Nurse Practitioner (n=9, 5)
|
4.4 outpatient visits
Standard Deviation 5.6
|
2.6 outpatient visits
Standard Deviation 2.2
|
|
Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers
Urologist (n=9, 1)
|
1.9 outpatient visits
Standard Deviation 1.3
|
2.0 outpatient visits
Standard Deviation NA
Data for standard deviation was not estimable as only 1 participant was analyzed for this measure.
|
|
Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers
Ophthalmology (n=5, 1)
|
1.0 outpatient visits
Standard Deviation 0.0
|
2.0 outpatient visits
Standard Deviation NA
Data for standard deviation was not estimable as only 1 participant was analyzed for this measure.
|
|
Healthcare Resource Use Survey: Number of Outpatient Visits to Healthcare Providers
Other (n=18, 6)
|
2.2 outpatient visits
Standard Deviation 2.8
|
1.0 outpatient visits
Standard Deviation 0.0
|
PRIMARY outcome
Timeframe: Baseline (Day 1)Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure.
Healthcare resources use survey of participants diagnosed with ATTR and caregivers was assessed by questions concerning a variety of different types of treatment and resources including outpatient visits to healthcare providers, hospitalizations, emergency/urgent care visits, symptomatic treatments, and out-of-pocket costs (for example, costs of travel to receive care).
Outcome measures
| Measure |
Participants Diagnosed With ATTR
n=9 Participants
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
n=6 Participants
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
|
|---|---|---|
|
Healthcare Resource Use Survey: Number of Hospitalizations
|
2.1 hospitalization visits
Standard Deviation 2.0
|
3.5 hospitalization visits
Standard Deviation 1.9
|
PRIMARY outcome
Timeframe: Baseline (Day 1)Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure.
Healthcare resources use survey of participants diagnosed with ATTR and caregivers was assessed by questions concerning a variety of different types of treatment and resources including outpatient visits to healthcare providers, hospitalizations, emergency/urgent care visits, symptomatic treatments, and out-of-pocket costs (for example, costs of travel to receive care).
Outcome measures
| Measure |
Participants Diagnosed With ATTR
n=13 Participants
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
n=6 Participants
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
|
|---|---|---|
|
Healthcare Resource Use Survey: Number of Emergency Care Visits
|
1.3 emergency care visits
Standard Deviation 0.9
|
2.8 emergency care visits
Standard Deviation 1.3
|
PRIMARY outcome
Timeframe: Baseline (Day 1)Population: All participants who were enrolled in the study and completed the survey.
Healthcare resources use survey of participants diagnosed with ATTR was assessed by questions concerning a variety of treatments and resources included outpatient visits to healthcare providers, hospitalizations, emergency/urgent care visits, symptomatic treatments, and out-of-pocket costs. Number of participants (diagnosed with ATTR) who visited non-medical practitioners (nutrition consultant/dietician, chiropractor, acupuncturist, massage therapist, occupational therapist or other than these) for symptomatic treatments were reported.
Outcome measures
| Measure |
Participants Diagnosed With ATTR
n=60 Participants
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
|
|---|---|---|
|
Healthcare and Resource Use Survey: Symptomatic Treatment of Participants
|
12 participants
|
—
|
PRIMARY outcome
Timeframe: Baseline (Day 1)Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure and 'n' signifies those participants who were evaluable for specific category.
Healthcare resources use survey of participants diagnosed with ATTR was assessed by questions concerning a variety of treatments and resources included outpatient visits to healthcare providers, hospitalizations, emergency/urgent care visits, symptomatic treatments, and out-of-pocket costs. Number of visits of participants (diagnosed with ATTR) who visited non-medical practitioners (nutrition consultant/dietician, chiropractor, acupuncturist, massage therapist, occupational therapist or other than these) for symptomatic treatments were reported.
Outcome measures
| Measure |
Participants Diagnosed With ATTR
n=12 Participants
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
|
|---|---|---|
|
Healthcare Resource Use Survey: Number of Symptomatic Treatment Visits
Nutrition Consultant/Dietitian (n=2)
|
4.0 symptomatic treatment visits
Standard Deviation 2.8 • Interval 2.0 to 6.0
|
—
|
|
Healthcare Resource Use Survey: Number of Symptomatic Treatment Visits
Chiropractor (n=1)
|
1.0 symptomatic treatment visits
Standard Deviation NA • Interval 1.0 to 1.0
Data for standard deviation was not estimable as only 1 participant was analyzed for this measure.
|
—
|
|
Healthcare Resource Use Survey: Number of Symptomatic Treatment Visits
Acupuncturist (n=3)
|
3.7 symptomatic treatment visits
Standard Deviation 2.5 • Interval 1.0 to 6.0
|
—
|
|
Healthcare Resource Use Survey: Number of Symptomatic Treatment Visits
Massage Therapist (n=5)
|
3.0 symptomatic treatment visits
Standard Deviation 1.9 • Interval 1.0 to 6.0
|
—
|
|
Healthcare Resource Use Survey: Number of Symptomatic Treatment Visits
Occupational Therapist (n=2)
|
3.5 symptomatic treatment visits
Standard Deviation 3.5 • Interval 1.0 to 6.0
|
—
|
|
Healthcare Resource Use Survey: Number of Symptomatic Treatment Visits
Other (n=3)
|
2.0 symptomatic treatment visits
Standard Deviation 1.0 • Interval 1.0 to 3.0
|
—
|
PRIMARY outcome
Timeframe: Baseline (Day 1)Population: All participants who were enrolled in the study and completed the survey. Here, 'n' signifies those participants who were evaluable for specific category.
Healthcare resources use survey of participants diagnosed with ATTR was assessed by questions concerning a variety of treatments and resources included outpatient visits to healthcare providers, hospitalizations, emergency/urgent care visits, symptomatic treatments, and out-of-pocket costs (expenditure on nutritional supplements, non-prescription medications and travel to receive medical care).
Outcome measures
| Measure |
Participants Diagnosed With ATTR
n=60 Participants
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
|
|---|---|---|
|
Healthcare Resource Use Survey: Out-of-Pocket Costs
Nutritional Supplements (n=13)
|
150 dollars
Full Range 121 • Interval 30.0 to 450.0
|
—
|
|
Healthcare Resource Use Survey: Out-of-Pocket Costs
Non-Prescription Medications (n=46)
|
28 dollars
Full Range 76 • Interval 0.0 to 300.0
|
—
|
|
Healthcare Resource Use Survey: Out-of-Pocket Costs
Travel to Receive Medical Care (n=45)
|
30 dollars
Full Range 823 • Interval 0.0 to 4000.0
|
—
|
PRIMARY outcome
Timeframe: Baseline (Day 1)Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure.
Participants diagnosed with ATTR rated their pain due to the health condition based on 3 items: pain right now, average pain in the past week, and worst pain in the past week prior to baseline visit. All 3 items were rated on an 11-point numeric rating scale ranging from 0=none to 10=severe pain, where higher scores indicated severe pain.
Outcome measures
| Measure |
Participants Diagnosed With ATTR
n=39 Participants
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
|
|---|---|---|
|
Participants Pain Score
Average Pain Past Week
|
3.4 units on a scale
Standard Deviation 3.0
|
—
|
|
Participants Pain Score
Pain Right now
|
3.1 units on a scale
Standard Deviation 3.1
|
—
|
|
Participants Pain Score
Worst Pain Past Week
|
4.3 units on a scale
Standard Deviation 3.4
|
—
|
PRIMARY outcome
Timeframe: Baseline (Day 1)Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure.
Norfolk QOL-DN: 35-item participant-rated questionnaire used to assess impact of neuropathy on the quality of life of participants diagnosed with ATTR. Scoring was based on 35 questions that yield a TQOL as well as 5 subscale scores: activities of daily living, large fiber neuropathy/physical functioning, small fiber neuropathy, autonomic neuropathy, and symptoms. TQOL= sum of all the items, total possible score range= -2 to 138, where higher score=worse quality of life. This outcome measure was planned to be analyzed only for the reporting arm of participants diagnosed with ATTR.
Outcome measures
| Measure |
Participants Diagnosed With ATTR
n=47 Participants
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
|
|---|---|---|
|
Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) Total Quality of Life (TQOL): Total Scores
|
54.6 units on a scale
Standard Deviation 32.8
|
—
|
PRIMARY outcome
Timeframe: Baseline (Day 1)Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure and 'n' signifies those participants who were evaluable for specific category.
Norfolk QOL-DN: 35-item participant-rated questionnaire used to assess impact of neuropathy on the quality of life of participants diagnosed with ATTR. It was summarized in 5 domains: (1) Activities of daily living (score ranges from 0 to 20, where higher score=worse quality of life); (2) Large fiber neuropathy/physical functioning (score ranges from -2 to 58, where higher score=worse condition); (3) Small fiber neuropathy (score ranges from 0 to 16, where higher score=worse condition); (4) Autonomic neuropathy (score ranges from 0 to 12, where higher score=worse condition) and (5) Symptoms (score ranges from 0 to 32, where higher score=less symptoms of disease). Total possible score range= -2 to 138, where higher score=worse quality of life. This outcome measure was analyzed only for the participants diagnosed with ATTR.
Outcome measures
| Measure |
Participants Diagnosed With ATTR
n=54 Participants
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
|
|---|---|---|
|
Norfolk Quality of Life-Diabetic Neuropathy Total Quality of Life: Subscale Scores
Symptoms (n= 50)
|
11.5 units on a scale
Standard Deviation 7.4
|
—
|
|
Norfolk Quality of Life-Diabetic Neuropathy Total Quality of Life: Subscale Scores
Activities of Daily Living (n= 50)
|
5.7 units on a scale
Standard Deviation 6.1
|
—
|
|
Norfolk Quality of Life-Diabetic Neuropathy Total Quality of Life: Subscale Scores
Small Fiber Neuropathy (n= 49)
|
6.0 units on a scale
Standard Deviation 4.2
|
—
|
|
Norfolk Quality of Life-Diabetic Neuropathy Total Quality of Life: Subscale Scores
Large Fiber Neuropathy (n= 47)
|
26.5 units on a scale
Standard Deviation 16.8
|
—
|
|
Norfolk Quality of Life-Diabetic Neuropathy Total Quality of Life: Subscale Scores
Autonomic Neuropathy (n= 50)
|
4.2 units on a scale
Standard Deviation 3.1
|
—
|
PRIMARY outcome
Timeframe: Baseline (Day 1)Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure.
KCCQ was a 23-item participant-completed questionnaire that assessed health status and health-related quality of life (HRQoL) in participants with heart failure. It was quantified in to following 10 summary scores: physical limitation, symptom frequency, symptom severity, and symptom stability, total symptoms, quality of life, social interference, self-efficacy, overall summary and clinical summary. Each summary score was scaled to range from 0 (minimum) to 100 (maximum), with higher scores representing greater disability. Total score ranged from 0 to 100, where higher scores indicated better functioning, fewer symptoms, and better disease specific quality of life.
Outcome measures
| Measure |
Participants Diagnosed With ATTR
n=17 Participants
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
|
|---|---|---|
|
Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores
Physical Limitation Score
|
41.7 units on a scale
Standard Deviation 29.2
|
—
|
|
Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores
Symptom Stability Score
|
48.5 units on a scale
Standard Deviation 10.7
|
—
|
|
Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores
Symptom Frequency Score
|
47.7 units on a scale
Standard Deviation 25.1
|
—
|
|
Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores
Symptom Burden Score
|
51.0 units on a scale
Standard Deviation 24.5
|
—
|
|
Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores
Total Symptom Score
|
49.3 units on a scale
Standard Deviation 23.8
|
—
|
|
Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores
Self-Efficacy Score
|
78.7 units on a scale
Standard Deviation 21.1
|
—
|
|
Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores
Quality of Life Score
|
50.0 units on a scale
Standard Deviation 18.6
|
—
|
|
Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores
Social Limitation Score
|
31.8 units on a scale
Standard Deviation 25.0
|
—
|
|
Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores
Overall Summary Score
|
43.2 units on a scale
Standard Deviation 20.0
|
—
|
|
Kansas City Cardiomyopathy Questionnaire (KCCQ) Scores
Clinical Summary Score
|
45.5 units on a scale
Standard Deviation 25.2
|
—
|
PRIMARY outcome
Timeframe: Baseline (Day 1)Population: All participants who were included in the study and completed the survey.
ZBI was a 22-item questionnaire designed to evaluate five broad aspects of caregiver burden in terms of personal and role strain associated with caregiving. Five broad aspects were: burden in the relationship, emotional well-being, social and family life, finances, loss of control over one's life. Each item rated on a 5 point scale anchored at 0 for "never" and 4 for "nearly always." Total score ranges from 0-88 with higher scores indicating increased burden of care.
Outcome measures
| Measure |
Participants Diagnosed With ATTR
n=32 Participants
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
|
|---|---|---|
|
Zarit Burden Interview (ZBI): Total Scores
|
28.8 units on a scale
Standard Deviation 17.5
|
—
|
PRIMARY outcome
Timeframe: Baseline (Day 1)Population: All participants who were included in the study and completed the survey. Here, 'n' signifies those participants who were evaluable for specific category.
A questionnaire designed to evaluate aspects of caregiver burden in terms of personal and role strain associated with caregiving. Total score of ZBI scale ranges from 0-88 with higher scores indicating increased burden of care. Five subscale scores were also calculated: (1) Burden in the relationship (consist of 6-items, ranging from 0 to 24 where higher scores indicating increased burden in relationship); (2) Emotional well-being (consisting of 7-items, ranging from 0 to 28 where higher scores indicating worse condition; (3) Social and family life (consisting of 4-items, ranging from 0 to 16 where higher scores indicating worse life condition); (4) Finances (consisting of a single item, scored from 0 to 4 where higher scores indicating worse financial condition); and (5) Loss of control over one's life (consisting of 4-items, ranging from 0 to 16 where higher scores indicating worse control over life).
Outcome measures
| Measure |
Participants Diagnosed With ATTR
n=32 Participants
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
|
|---|---|---|
|
Zarit Burden Interview: Subscale Scores
Burden in Relationship (n=28)
|
7.6 units on a scale
Standard Deviation 5.0
|
—
|
|
Zarit Burden Interview: Subscale Scores
Emotional Well-Being (n=29)
|
8.3 units on a scale
Standard Deviation 5.9
|
—
|
|
Zarit Burden Interview: Subscale Scores
Social and Family Life (n=28)
|
5.1 units on a scale
Standard Deviation 3.4
|
—
|
|
Zarit Burden Interview: Subscale Scores
Finances (n=29)
|
1.9 units on a scale
Standard Deviation 1.4
|
—
|
|
Zarit Burden Interview: Subscale Scores
Loss of Control Over Life (n=29)
|
6.5 units on a scale
Standard Deviation 3.5
|
—
|
PRIMARY outcome
Timeframe: Baseline (Day 1)Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure.
Caregivers completed a series of questions related to the number of hours per week spent on providing care and support to the participants diagnosed with ATTR.
Outcome measures
| Measure |
Participants Diagnosed With ATTR
n=29 Participants
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
|
|---|---|---|
|
Caregiver Burden Items Assessment: Number of Hours Per Week Spent in Care of the Participants With ATTR
|
72 hours per week
Interval 2.0 to 168.0
|
—
|
PRIMARY outcome
Timeframe: Baseline (Day 1)Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure.
Caregivers completed a series of questions related to the loss in their working time while providing care and support to the participants diagnosed with ATTR.
Outcome measures
| Measure |
Participants Diagnosed With ATTR
n=26 Participants
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
|
|---|---|---|
|
Caregiver Burden Items Assessment: Work Time Lost
|
22 hours
Interval 0.0 to 168.0
|
—
|
PRIMARY outcome
Timeframe: Baseline (Day 1)Population: All participants who were enrolled in the study and completed the survey. Here, 'N' signifies those participants who were evaluable for this measure.
Caregivers completed a series of questions related to the total cost spent on providing healthcare support to participants diagnosed with ATTR.
Outcome measures
| Measure |
Participants Diagnosed With ATTR
n=20 Participants
Participants diagnosed with transthyretin amyloidosis (ATTR) with two phenotypes: familial amyloid polyneuropathy (TTR-FAP) and cardiac amyloidosis (TTR-CM) were included in this non-interventional study to complete the one time survey.
|
Caregivers
Caregivers were those participants who provided support and care to the participants diagnosed with ATTR (TTR-FAP and TTR-CM) and completed the caregiver survey, regardless of whether they themselves were diagnosed with ATTR or not.
|
|---|---|---|
|
Caregiver Burden Items Assessment: Total Cost
|
9313.0 dollars
Standard Deviation 33226.1 • Interval 0.0 to 150000.0
|
—
|
Adverse Events
Participants Diagnosed With ATTR
Caregivers
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
- Publication restrictions are in place
Restriction type: OTHER